172 Participants Needed

Omaveloxolone for Friedreich's Ataxia

Recruiting at 10 trial locations
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

This trial is testing omaveloxolone, a drug that may help people with Friedreich's ataxia by boosting their body's natural defenses against cell damage. The study focuses on patients with this genetic condition because they have weakened antioxidant systems. Omaveloxolone works by activating a protein that helps protect cells from damage. It aims to improve cell function and reduce harmful effects in people with Friedreich's ataxia.

Do I need to stop my current medications for the trial?

You may need to stop taking certain medications before joining the trial. Specifically, you cannot take certain drugs that affect liver enzymes or are processed by specific pathways in the body within 7 days before starting the study. It's best to discuss your current medications with the study team to see if any need to be stopped.

What data supports the effectiveness of the drug Omaveloxolone for Friedreich's Ataxia?

Omaveloxolone has been shown to activate the Nrf2 pathway, which helps reduce oxidative stress and improve mitochondrial function in Friedreich's Ataxia, a condition with no approved treatments. Clinical trials have demonstrated its potential benefits in improving symptoms by targeting dysfunctional pathways in the disease.12345

Is omaveloxolone safe for humans?

Omaveloxolone has been studied for safety in patients with Friedreich's ataxia, and it has been approved in the USA for this condition, suggesting it is generally considered safe for human use in this context.12345

How is the drug omaveloxolone unique in treating Friedreich's ataxia?

Omaveloxolone is unique because it activates the Nrf2 pathway, which helps reduce oxidative stress and improve mitochondrial function, addressing key issues in Friedreich's ataxia. Unlike other treatments, it is an oral medication specifically approved for this condition, offering a novel approach by targeting the underlying cellular dysfunction.12345

Eligibility Criteria

This trial is for people aged 16-40 with genetically confirmed Friedreich's ataxia, who can swallow capsules and have a specific level of disease severity (modified FARS score between 20 and 80). Participants should be stable in their exercise routine and able to complete maximal exercise testing. They cannot join if they've used certain drugs recently, have significant heart disease or abnormal blood tests, uncontrolled diabetes, cognitive impairments, active infections, or substance abuse issues.

Inclusion Criteria

My condition is genetically confirmed Friedreich's ataxia.
I am between 16 and 40 years old.
Have no changes to exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the 16-week study period
See 3 more

Exclusion Criteria

I have not taken and will not take certain drugs that affect liver enzymes or drug transporters during the study.
Have participated in any other interventional clinical study within 30 days prior to Study Day 1
My diabetes is not under control (HbA1c >11.0%).
See 8 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Randomized, placebo-controlled, double-blind, dose-escalation study to evaluate the safety of omaveloxolone (RTA 408) at various doses

12 weeks

Treatment Part 2

Randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety and efficacy of omaveloxolone (RTA 408) 150 mg

48 weeks

Extension

Long-term safety and tolerability of omaveloxolone (RTA 408) in qualified participants, open-label

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Omaveloxolone
Trial Overview The study examines omaveloxolone (RTA 408) capsules at various doses for treating Friedreich's ataxia. It has two parts: the first part tests safety across different doses; the second part assesses safety and effectiveness of a set dose (150 mg). An extension phase will look into long-term use. The drug aims to activate Nrf2 signaling impaired in this condition.
Participant Groups
10Treatment groups
Experimental Treatment
Placebo Group
Group I: Part 2 Omaveloxolone Capsules 150 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 150 mg administered orally once daily for 48 weeks
Group II: Part 1 Omaveloxolone Capsules 80 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 80 mg administered orally once daily for 12 weeks
Group III: Part 1 Omaveloxolone Capsules 40 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 40 mg administered orally once daily for 12 weeks
Group IV: Part 1 Omaveloxolone Capsules 300 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 300 mg administered orally once daily for 12 weeks
Group V: Part 1 Omaveloxolone Capsules 20 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 20 mg administered orally once daily for 12 weeks
Group VI: Part 1 Omaveloxolone Capsules 2.5 and 5 mgExperimental Treatment2 Interventions
omaveloxolone (RTA 408) Capsules, 2.5 mg administered orally one daily for 2 weeks, then 5 mg taken orally once daily for 10 weeks
Group VII: Part 1 Omaveloxolone Capsules 160 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 160 mg administered orally once daily for 12 weeks
Group VIII: Part 1 Omaveloxolone Capsules 10 mgExperimental Treatment1 Intervention
omaveloxolone (RTA 408) Capsules, 10 mg administered orally once daily for 12 weeks
Group IX: Part 1 Placebo CapsulesPlacebo Group1 Intervention
Placebo capsules administered orally once daily for 12 weeks
Group X: Part 2 Placebo CapsulesPlacebo Group1 Intervention
Placebo capsules administered orally once daily for 48 weeks

Omaveloxolone is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Skyclarys for:
  • Friedreich's ataxia
🇪🇺
Approved in European Union as Skyclarys for:
  • Friedreich's ataxia

Find a Clinic Near You

Who Is Running the Clinical Trial?

Biogen

Lead Sponsor

Trials
655
Recruited
468,000+
Daniel Quirk profile image

Daniel Quirk

Biogen

Chief Medical Officer

MD

Christopher A. Viehbacher profile image

Christopher A. Viehbacher

Biogen

Chief Executive Officer since 2022

Graduated from Queen's University, Kingston, Ontario, Canada

Reata, a wholly owned subsidiary of Biogen

Lead Sponsor

Trials
52
Recruited
7,400+

Reata Pharmaceuticals, Inc.

Lead Sponsor

Trials
52
Recruited
6,900+

Friedreich's Ataxia Research Alliance

Collaborator

Trials
20
Recruited
6,400+

AbbVie

Industry Sponsor

Trials
1,079
Recruited
535,000+
Founded
2013
Headquarters
North Chicago, USA
Known For
Immunology treatments
Top Products
Humira (adalimumab), Skyrizi (risankizumab), Rinvoq (upadacitinib)

Dr. Roopal Thakkar

AbbVie

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine

Robert A. Michael profile image

Robert A. Michael

AbbVie

Chief Executive Officer

Bachelor's degree in Finance from the University of Illinois

Findings from Research

Omaveloxolone (SKYCLARYS™) is an orally active drug that enhances antioxidant activity by activating the Nrf2 pathway, which is crucial for combating oxidative stress and mitochondrial dysfunction in patients with Friedreich's ataxia.
Approved in February 2023, omaveloxolone represents a significant advancement in treating Friedreich's ataxia, particularly for adults and adolescents aged 16 and older, by targeting the underlying cellular damage associated with the disease.
Omaveloxolone: First Approval.Lee, A.[2023]
Omaveloxolone, a synthetic compound that activates the Nrf2 pathway, showed a dose-linear pharmacokinetic profile in monkeys, indicating predictable absorption and distribution in key tissues like the liver, lung, and brain after oral administration.
In Friedreich's ataxia patients, doses of omaveloxolone starting at 80 mg led to significant improvements in neurological function, with plasma levels correlating with those that effectively activated Nrf2 in monkeys, suggesting its potential efficacy in treating this condition.
Pharmacokinetics and pharmacodynamics of the novel Nrf2 activator omaveloxolone in primates.Reisman, SA., Gahir, SS., Lee, CI., et al.[2022]
In a phase 2 trial involving 103 patients with Friedreich ataxia, omaveloxolone significantly improved neurological function compared to placebo, with a notable difference in modified Friedreich's Ataxia Rating Scale scores after 48 weeks.
Omaveloxolone was generally safe and well tolerated, although some patients experienced transient increases in liver enzymes and common side effects like headache, nausea, and fatigue.
Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study).Lynch, DR., Chin, MP., Delatycki, MB., et al.[2023]

References

Omaveloxolone: First Approval. [2023]
Pharmacokinetics and pharmacodynamics of the novel Nrf2 activator omaveloxolone in primates. [2022]
Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study). [2023]
Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia. [2022]
Omaveloxolone: potential new agent for Friedreich ataxia. [2021]
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